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Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD.
(academic.oup.com)
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Nucleic Acids Research 2026-04-13 | #therapeutics #gene therapy #muscular dystrophy #rnai #drug delivery | |
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Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency.
(academic.oup.com)
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Nucleic Acids Research 2026-04-13 | #therapeutics #rnai #off-target #nucleic acid modification |